Pharmacogenetics
The cost of clinical trials
3.13 The costs of developing a new medicine are substantial.6 The development of a new medicine takes an average of 10–15 years (see Box 3.1). Currently, only around 2% of all programmes to develop new medicines result in clinical trials. Of the compounds that do reach the stage of Phase I testing, only 20% will eventually be approved for clinical use.7 The effect of pharmacogenetics on the cost of clinical trials and therefore of new medicines is difficult to predict at this stage, though a number of speculative papers have been published on the topic.8
3.14 The selection of smaller groups of genetically homogeneous participants in clinical trials may be advantageous, leading to more robust and reliable scientific findings about the group of patients for whom the medicine might eventually be prescribed. However, there are various reasons to be cautious about claims that clinical trials will be reduced in size and therefore cost. First, larger numbers of patients may be needed in Phase II trials in order to identify relevant pharmacogenetic variants, since these variants may be relatively rare.
Secondly, in order to identify adverse reactions, a large number of patients may still be required, since many reactions, including some of the most problematic, are relatively infrequent. This could mean that the numbers of participants in the later stages of clinical trials cannot easily be reduced, or that extended Phase IV monitoring is required. Thirdly, participants will still be required to take the medicine for similar periods of time as at present, in order to generate statistically significant information about its effects. Fourthly, costs may be increased because of expenditure on pharmacogenetic tests and analysis of the data they produce. Finally, it has been suggested that the cost of trials may increase because it would take longer to identify and recruit sufficient numbers of genetically similar participants. However, a counterbalance may be that the trials themselves, in which medicines targeted to the specific group of participants are tested, might produce results more quickly.
3.15 A number of respondents to the Working Party’s consultation suggested that pharmacogenetics could be expected to increase the cost of clinical trials in the short term, but could contribute to a reduction in the long term: ‘the economic impact of pharmacogenetics on the development of new medicines is difficult to gauge at present although in the short term costs may be higher. However many authors have cited the potential for pharmacogenetics to make clinical trials shorter and smaller thus reducing costs in development in the long term.’ (European Federation of Pharmaceutical Industries and Associations and Association of the British Pharmaceutical Industry)
While the effect of pharmacogenetics may be to reduce some of the costs of developing new medicines, it would be imprudent to infer from this that the cost of purchasing medicines will necessarily fall. At this stage, it is not possible to predict the impact of pharmacogenetics on the cost of medicines.
Footnotes6 The Tufts Center for the Study of Drug Development has estimated the cost of developing a new prescription medicine at $897 million (Kaitin K (2003) Post-approval R&D raises total drug development costs to $897 million, Tufts Center for the Study of Drug Development Impact Report May/Jun; 5(3)). However, the US national consumer group Public Citizen has suggested that the true costs are significantly lower (see Public Citizen (2001) New Study Expected to Significantly Overstate Drug Industry R&D Costs. Available: http://www.citizen.org/pressroom/release.cfm?ID=942. Accessed on: 31 Oct 2002.) See also Relman AS and Angell M (2002) America’s other drug problem, in The New Republic, December 16, pp. 27-41.
7 Pharmaceutical Research and Manufacturers of America (2003) Pharmaceutical Industry Profile 2003 (Washington, DC: PhRMA).
8 Tollman P et al. (2001) A Revolution in R&D (Boston: Boston Consulting Group). This paper suggests that pharmacogenetics will reduce the cost of developing new medicines. The savings would be achieved by refining trials, reducing the number of participants, and by bringing to market medicines that would otherwise have been lost because they are only effective in a subset of patients with a particular disease. See also Peakman T and Arlington S (2001) Putting the Code to Work: The Promise of Pharmacogenetics and Pharmacogenomics (PricewaterhouseCoopers).